MSA is a Parkinsonian disease that causes damage to nerve cells. Image credit: Shutterstock/Andrii Vodolazhskyi.

The US Food and Drug Administration (FDA) has granted Inhibikase Therapeutics’ risvodetinib orphan drug designation, speeding up a potential therapy for multiple system atrophy (MSA).

In March 2023, Atlanta, US-headquartered Inhibikase received investigational new drug (IND) clearance for its planned Phase II studies. In a statement, Inhibikase CEO Dr Milton Werner said further updates on the trial will be announced in the coming quarters.

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At the Movement Disorder Society (MDS) Congress in Copenhagen, Denmark held in August 2023, the pharma company presented preclinical data demonstrating the therapeutic activity of the candidate in MSA.

Inhibikase’s pipeline focuses on neurodegeneration. Risvodetinib is its lead candidate and is an Abelson Tyrosine Kinase (c-Abl) inhibitor. In 2022, Inhibikase published findings indicating that c-Abl is activated in the brains of MSA patients and has a key role in the disease process via its modification of alpha-synuclein aggregates.

MSA is a Parkinsonian disease which affects the central and autonomic nervous systems, meaning movement and involuntary functions are impacted. The progressive neurodegenerative disorder advances rapidly, with patients becoming bedbound around seven to 10 years after the first symptoms appear.

The orphan drug designation includes benefits such as tax credits on clinical trials and potential market exclusivity after approval.

Dr Werner said: “There are currently no approved symptomatic or disease-modifying therapies for MSA on the market. As we look ahead, we are advancing our ongoing animal model studies of risvodetinib to determine its therapeutic potential to block progression and correct functional loss in MSA.”

“These studies will form the basis of our planned Phase II clinical study. We believe proof that risvodetinib is clinically beneficial in MSA will form a basis for potential success in other forms of Parkinsonism,” noted Dr. Milton Werner, Chief Executive of Inhibikase Therapeutics.”

In March 2023, Australian biotech Alterity Therapeutics began dosing in its Phase II trial investigating its α-synuclein pathology inhibiting candidate. Alterity’s ATH434 has also received orphan drug designation from the FDA.